PepGen develops Enhanced Delivery Oligonucleotides (EDOs) to treat neuromuscular and neurologic diseases, using peptide-conjugated antisense oligonucleotides to improve delivery to multiple muscle tissues including cardiac tissue. Its lead DMD program, EDO51, aims to induce exon 51 skipping and is planned to enter Phase 1 trials in 2022; preclinical data suggest higher exon-skipping and dystrophin production at lower doses than existing therapies. The lead DM1 program, EDODM1, is designed to block toxic CUG repeats and is expected to enter clinical trials in early 2023. PepGen plans to expand its DMD program to additional exon-skipping populations and build a broader pipeline of oligonucleotide candidates for neuromuscular and neurologic diseases. The company is headquartered in Boston and will use recent proceeds to advance programs into the clinic and expand its team. Financially, PepGen announced an oversubscribed $112.5 million crossover financing following a $45 million Series A in December 2020. PepGen develops next-generation cell-penetrating peptides conjugated to phosphorodiamidate morpholino oligomers (PPMOs) to improve delivery and safety of oligonucleotide therapies. The platform is designed to enhance tissue distribution, notably achieving strong cardiac penetration unlike some competing approaches. Lead programs target Duchenne muscular dystrophy (DMD) and a pipeline of rare neuromuscular, neurologic, and primary cardiac conditions with severe cardiac manifestations. The company aims to advance its PPMO candidates into the clinic using the newly raised capital. PepGen emphasizes improved cellular uptake while engineering out prior safety signals associated with peptide delivery approaches. Its corporate headquarters will be in Boston while its research hub remains in the UK.

Total raised

$158M

Funding rounds

2

Latest round

Equity

Latest activity

Aug 2021